The executive director of the Spanish Association of Orphan and Ultra-Orphan Drug Laboratories (AELMHU), Marian Corral, participated this Tuesday in the “Multidisciplinary Conference on Rare Diseases, ” held at the Gregorio Marañón University General Hospital, where she discussed the main challenges and advances in the development of and access to treatments for these conditions.
The event, which was sponsored by CSL, Kyowa Kirin, Sanofi and Sobihas brought together experts from the healthcare sector, industry representatives, and patient associations, with the aim of establishing itself as a platform for multidisciplinary dialogue focused on improving the management of rare diseases.
In her remarks, Marian Corral provided an overview of the journey that orphan and ultra-orphan drugs take, from the initial stages of research to their eventual delivery to patients. In this regard, she emphasized the complexity of researching rare diseases, many of which are still poorly understood scientifically, which makes it difficult to identify therapeutic targets and develop effective treatments.
The director of AELMHU also highlighted the significant investment in research and development (R&D) that the pharmaceutical industry is making in this area. As she explained, these are lengthy, technically complex processes involving a high degree of uncertainty, which requires a sustained commitment from the companies involved.
On the regulatory front, Corral highlighted the role of the European regulatory framework, which provides specific incentives to promote innovation in medicines for rare diseases. These measures, he noted, have been key to driving progress in this area.
However, he also pointed out the ongoing challenges regarding access and funding in Spain. In particular, he emphasized the need to improve coordination among the various levels of the healthcare system to ensure that patients can access these treatments on an equitable basis and within a reasonable timeframe.
